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Prepublished online as a Blood First Edition Paper on October 10, 2002; DOI 10.1182/blood-2002-04-1203.

Submitted April 22, 2002
Accepted September 27, 2002
Transplantation of highly purified CD34+ progenitor cells from unrelated donors in pediatric leukemia
Peter Lang, Rupert Handgretinger*, Dietrich Niethammer, Paul G Schlegel, Michael Schumm, Johann Greil, Peter Bader, Corinna Engel, Hans Scheel-Walter, Matthias Eyrich, and Thomas Klingebiel
Children's University Hospital, University of Tuebingen, Tuebingen, Germany
St. Jude Children's Research Hospital, Memphis, TN, USA
Children's University Hospital, University of Wuerzburg, Wuerzburg, Germany
Institute for Biostatistics, University of Tuebingen, Tuebingen, Germany
Children's University Hospital, University of Frankfurt, Frankfurt am Main, Germany
* Corresponding author; email: rupert.handgretinger{at}stjude.org.
Unrelated donors are commonly used for hematopoietic stem cell transplants, but graft versus host disease (GvHD) is a major problem. We investigated whether transplantation of purified mobilized peripheral-blood CD34+ stem cells from unrelated donors would prevent acute and chronic GvHD in pediatric patients with leukemia and avert the need for pharmacological immunosuppression. Thirty-one pediatric patients with acute lymphoblastic leukemia (ALL, n=16), acute myeloid (n=7), chronic myeloid (n=6), or juvenile myelomonocytic leukemia (n=2) underwent transplantation. The median purity of CD34+ cells after positive magnet-activated cell sorting was 98.5%. Patients received a median of 8.0 x 106 CD34+ cells and 6 x 103 CD3+ T lymphocytes per kilogram, with no posttransplant pharmacological immunosuppression. Primary acute GvHD grade 2 was seen in only 10% of patients (n=3) and occurred only after HHV 6 infection. Two patients had limited chronic GvHD. Engraftment occurred in all patients (primary engraftment, n=26; engraftment after reconditioning, n=5). The 2-year survival estimate was 38% for all patients and 63% for patients with ALL in complete remission. Patients with myeloid malignancies had a poor outcome. In comparison to a historical control group that received unmanipulated bone marrow, our patients had a lower incidence of GvHD (p <0.001). No difference was observed in the probability of relapse or survival. Study patients with ALL in remission showed a trend toward better survival (p =0.07). Transplantation of purified peripheral-blood CD34+ cells from unrelated donors effectively minimizes GvHD and may be a good therapeutic option for patients with relapsed ALL.

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